First human trial tests One-Shot gene therapy for rare genetic disorder
NCT ID NCT06662188
Summary
This early-stage study is testing a single dose of an experimental gene therapy called JAG201 in children aged 2-9 with SHANK3 deficiency, which causes Phelan-McDermid Syndrome. The main goal is to check if the treatment is safe and tolerable when injected into the fluid around the brain. Researchers will also look for any early signs that it might help improve developmental symptoms.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Rush University
RECRUITINGChicago, Illinois, 60612, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Seaver Autism Center at Mount Sinai
RECRUITINGNew York, New York, 10029, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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